VALENZANO, KENNETH JOSEPH,CROWLEY, JOHN,KHANNA, RICHIE,FLANAGAN, JOHN
申请号:
CA2866683
公开号:
CA2866683A1
申请日:
2013.03.07
申请国别(地区):
CA
年份:
2013
代理人:
摘要:
The present application provides for compositions comprising high concentrations of acid a-glucosidase in combination with an active site-specific chaperone for the acid a-glucosidase, and methods for treating Pompe disease in a subject in need thereof, that includes a method of administering to the subject such compositions. The present application also provides methods for increasing the in vitro and in vivo stability of an acid a-glucosidase enzyme formulation.